Gene therapy with AR isoform 2 rescues spinal and bulbar muscular atrophy phenotype by modulating AR transcriptional activity

Lim, Wooi F Forouhan, Mitra Roberts, Thomas C Dabney, Jesse Ellerington, Ruth Speciale, Alfina A Manzano, Raquel Lieto, Maria Sangha, Gavinda Banerjee, Subhashis Conceição, Mariana Cravo, Lara Biscans, Annabelle Roux, Loïc Pourshafie, Naemeh Grunseich, Christopher Duguez, Stephanie Khvorova, Anastasia Pennuto, Maria Cortes, Constanza J La Spada, Albert R Fischbeck, Kenneth H Wood, Matthew JA Rinaldi, Carlo Gene therapy with AR isoform 2 rescues spinal and bulbar muscular atrophy phenotype by modulating AR transcriptional activity

Sci. adv.

10.1126/sciadv.abi6896 2021

2021